uniQure Advancing AMT-130 Development in the New Year


In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.

In 2018, uniQure N.V. expects to advance the clinical development of AMT-130, its investigational gene therapy for the treatment of Huntington’s disease (HD).

The company has stated its intentions to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) in the second half of the year. The first human clinical trial with the drug should begin soon after the FDA’s projected acceptance.

HD is a genetic, neurodegenerative disorder that leads to loss of muscle coordination, behavioral abnormalities and cognitive decline. It usually begins to present when a person turns 30-to-40 years old. The gene therapy AMT-130 was granted Orphan Drug Designation by the FDA in October and is a Huntingtin-lowering approach that would be mediated through viral delivery of adeno-associated virus (AAV).

“We ended 2017 with significant momentum across all of our programs and the cash to fund operations into 2020,” stated Matthew Kapusta, chief executive officer of uniQure, in a press release. “Now that we have established robust, commercial-scale manufacturing in our Lexington facility, we are highly focused on the near-term initiation of our pivotal study of AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in Huntington’s disease.”

The next step in the clinical development of AMT-130 centers around the initiation of a Phase 1/2 study for the HD indication. The company plans to complete its Good Laboratory Practices (GLP) toxicology study of AMT-130 and make the drug the first AAV-based gene therapy to begin human evaluation.

“We also are intensifying efforts to further expand our gene therapy product pipeline,” said Kapusta. “In addition to AMT-126, our BMS-partnered gene therapy candidate for congestive heart failure, we have a number of promising early-stage projects focused on liver-directed and CNS disorders. We expect multiple new INDs to be filed over the next two years, further leveraging our industry-leading manufacturing and technology platform.”

At present, treatment options for HD patients are limited to alleviation from symptoms, but they do not delay the onset or slow progression. It is widely believed that AMT-130 has the potential to completely stop the disease.

At the European Society of Gene and Cell Therapy (ESGCT) Congress in Berlin, Germany last October, uniQure presented clinical data showing the effectiveness of AMT-130 in an R6/2 mouse model, exhibiting its ability to improve motor symptoms and survival. Additionally, the study observed that the gene therapy did alter the biochemistry of the brain with a significant reduction in the expression of the mutant Huntingtin protein.

In addition to AMT-130, uniQure is developing its gene therapy pipeline for other diseases: AMT-12 for congestive heart failure, and AMT-061 for hemophilia B.

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